Gene therapy for primary immunodeficiencies: part 1
Highlights ► Gene therapy provided a clinical benefit to most of the 60 patients affected by ADA-deficiency or SCID-X1. ► Gene therapy can be a valuable alternative to allogeneic hematopoietic stem cell transplantation. ► Insertional oncogenesis still represents the major issue to solve. ► Homologou...
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| Veröffentlicht in: | Current opinion in immunology 2012-10, Vol.24 (5), p.580-584 |
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| container_end_page | 584 |
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| container_issue | 5 |
| container_start_page | 580 |
| container_title | Current opinion in immunology |
| container_volume | 24 |
| creator | Cavazzana-Calvo, Marina Fischer, Alain Hacein-Bey-Abina, Salima Aiuti, Alessandro |
| description | Highlights ► Gene therapy provided a clinical benefit to most of the 60 patients affected by ADA-deficiency or SCID-X1. ► Gene therapy can be a valuable alternative to allogeneic hematopoietic stem cell transplantation. ► Insertional oncogenesis still represents the major issue to solve. ► Homologous recombination is rapidly improving to edit gene mutations. |
| doi_str_mv | 10.1016/j.coi.2012.08.008 |
| format | Article |
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| source | MEDLINE; Elsevier ScienceDirect Journals Complete |
| subjects | Adenosine Deaminase - deficiency Adenosine Deaminase - genetics Allergy and Immunology Animals Clinical Trials as Topic - methods Clinical Trials as Topic - trends Disease Models, Animal Genetic Therapy - methods Genetic Therapy - trends Hematopoietic Stem Cell Transplantation - methods Hematopoietic Stem Cell Transplantation - trends Humans Immunologic Deficiency Syndromes - genetics Immunologic Deficiency Syndromes - immunology Immunologic Deficiency Syndromes - therapy X-Linked Combined Immunodeficiency Diseases - genetics X-Linked Combined Immunodeficiency Diseases - immunology X-Linked Combined Immunodeficiency Diseases - therapy |
| title | Gene therapy for primary immunodeficiencies: part 1 |
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